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allogeneic hematopoietic stem cells (University of Illinois at Chicago)

Development stage
Unknown
Lead developer
National Institutes of Health
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

This is an investigational allogeneic hematopoietic stem cell transplantation (HSCT) protocol developed by the University of Illinois at Chicago (UIC) specifically for the treatment of adults with high-risk or aggressive sickle cell disease (SCD). The therapy involves the infusion of HLA-matched related donor hematopoietic stem cells following a non-myeloablative conditioning regimen. This regimen consists of the monoclonal antibody alemtuzumab (targeting CD52) and low-dose total body irradiation (300 cGy), designed to achieve stable mixed chimerism—a state where donor and recipient cells coexist—without the severe toxicity associated with traditional high-dose myeloablative chemotherapy. Post-transplant, patients receive sirolimus for immunosuppression. The primary goal is to replace the recipient's defective erythropoiesis with healthy donor cells to reverse the sickle cell phenotype while minimizing transplant-related morbidity and mortality.

Other names
Alemtuzumab-based non-myeloablative allogeneic hematopoietic stem cell transplantationUIC sickle cell transplant protocolAllogeneic Stem Cell Transplant for Aggressive Sickle Cell Disease

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